8:00 am
Registration & Breakfast

8:50 am Chair’s Opening Remarks


9:00 am Panel Discussion: The Challenges Associated With Scaling-Up to Commercial Manufacturing

  • Anne Douar Chief Development Officer, Vivet Therapeutics
  • Niklas Engler Head Cell and Gene Therapy Strategy, Roche
  • Markus Haindl Global Head Of Gene Therapy & Technical Development, Roche


  • Personal experiences in building gene therapy manufacturing facilities
  • Weighing up the choice between in-house manufacturing and outsourcing to CMOs
  • Considerations for deciding between scaling up and scaling out
  • Understanding how to demonstrate comparability when scaling up manufacturing processes
  • Integrating technology into modern gene therapy manufacturing approaches

9:30 am Developing Gene Therapy Manufacturing Processes to Overcome Productivity Bottlenecks

  • Markus Haindl Global Head Of Gene Therapy & Technical Development, Roche


  • How the use of technology can boost manufacturing productivity
  • Successful CMC techniques to streamline the clinical development life cycle
  • Top tips for successfully demonstrating manufacturing comparability when preparing to launch the product into market

10:00 am Reserved for THREAD Research

10:30 am Morning Refreshments & Speed Networking


Our speed networking is the ideal opportunity to get face-to-face time with colleagues and peers who are passionate about overcoming regulatory challenges to bring gene therapies to those most in need. Introduce yourself to the attendees that you would like to have more in-depth conversations with, explore common challenges, and establish meaningful relationships to pursue for the rest of the conference and beyond.

11:30 am Highlighting How to Best Demonstrate Comparability in Your Manufacturing Processes

  • Niamh Kinsella Global Regulatory CMC Early Development Gene Therapy Lead, Biogen


  • What is comparability and how does it impact the overall development plan of the product?
  • How to demonstrate comparability to regulators during development
  • Best practices: how to prepare early to meet authority expectations

12:00 pm Outlining The Importance of Achieving Consistency & Minimising Variability in Gene Therapy Manufacturing Processes


  • Top strategies to address process variability challenges
  • Understanding the importance of selecting the correct raw materials
  • Technical development strategies to support successful clinical development

12:30 pm Reserved for The Jackson Laboratory

1:00 pm
Lunch & Networking


2:00 pm Panel Discussion: Clinical Development Processes for Rare Disorders: Moving Towards a Patient-Driven Approach

  • Silvia Cerolini Founder & Chief Executive Officer, Eyes on the Future
  • Nick Sireau Board Chair & Chief Executive Officer, AKU Society
  • Rick Thompson Chief Executive Officer, Beacon for Rare Diseases


  • The unique challenges faced in attaining treatments by patients with rare disorders
  • Where patient perspectives can be conducive to success in the clinical trial endpoint design of therapies for rare disorders
  • Good and bad clinical development stories from trials taking different approaches to patient advocacy input
  • The case behind biotech and pharma incorporating patient perspective early in their drug development processes

2:45 pm Lumevoq Gene Therapy in Leber Hereditary Optic Neuropathy: Key Learnings in Clinical Trial Design


  • The unique challenges in clinical trial design for acute rare disease
  • Reviewing the use of indirect comparison versus natural history data
  • How to simultaneously optimise clinical development processes whilst answering to regulatory requirements

3:15 pm
Afternoon Refreshments

3:45 pm Maintaining Perspective: Aligning the Clinical Trial Endpoints With the Needs of the Patient


  • How attitudes towards risk-benefit vary between patients, drug developers and regulators
  • Exploring the need to harmonise these viewpoints
  • The importance of presenting patient and caregiver perspectives to the regulators

4:15 pm Leveraging Regulatory Scientific Advice for Translational Success From Preclinical to Clinical and Beyond


  • How early seeking of scientific advice can help pre-empt challenges transitioning into clinical development
  • Why this open dialogue can be mutually beneficial for both the developers and the regulators
  • The complexities drug developers are facing navigating supply-chain challenges

4:45 pm Chair’s Closing Remarks